BR112019024256A2 - composição de ácido nucleico, método para tratar hemofilia b e para produzir uma partícula de vírus adenoassociado, e, uso de uma composição de ácido nucleico. - Google Patents
composição de ácido nucleico, método para tratar hemofilia b e para produzir uma partícula de vírus adenoassociado, e, uso de uma composição de ácido nucleico. Download PDFInfo
- Publication number
- BR112019024256A2 BR112019024256A2 BR112019024256A BR112019024256A BR112019024256A2 BR 112019024256 A2 BR112019024256 A2 BR 112019024256A2 BR 112019024256 A BR112019024256 A BR 112019024256A BR 112019024256 A BR112019024256 A BR 112019024256A BR 112019024256 A2 BR112019024256 A2 BR 112019024256A2
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- Brazil
- Prior art keywords
- nucleic acid
- acid composition
- producing
- treating hemophilia
- virus particle
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
- A61K38/482—Serine endopeptidases (3.4.21)
- A61K38/4846—Factor VII (3.4.21.21); Factor IX (3.4.21.22); Factor Xa (3.4.21.6); Factor XI (3.4.21.27); Factor XII (3.4.21.38)
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/66—Microorganisms or materials therefrom
- A61K35/76—Viruses; Subviral particles; Bacteriophages
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
- A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/745—Blood coagulation or fibrinolysis factors
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/62—DNA sequences coding for fusion proteins
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2800/00—Nucleic acids vectors
- C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- Medicinal Chemistry (AREA)
- Zoology (AREA)
- Biochemistry (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Epidemiology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Virology (AREA)
- Hematology (AREA)
- Toxicology (AREA)
- Diabetes (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Immunology (AREA)
- Mycology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Peptides Or Proteins (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Abstract
a presente divulgação fornece, entre outros aspectos, polinucleotídeos com alteração de códon que codificam variantes de fator ix para expressão em células de mamífero. em algumas modalidades, a divulgação também fornece vetores de terapia gênica de mamífero e métodos para tratar hemofilia b.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201762509616P | 2017-05-22 | 2017-05-22 | |
PCT/US2018/033866 WO2018217731A1 (en) | 2017-05-22 | 2018-05-22 | Viral vectors encoding recombinant fix with increased expression for gene therapy of hemophilia b |
Publications (1)
Publication Number | Publication Date |
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BR112019024256A2 true BR112019024256A2 (pt) | 2020-06-09 |
Family
ID=62621009
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
BR112019024256A BR112019024256A2 (pt) | 2017-05-22 | 2018-05-22 | composição de ácido nucleico, método para tratar hemofilia b e para produzir uma partícula de vírus adenoassociado, e, uso de uma composição de ácido nucleico. |
Country Status (17)
Country | Link |
---|---|
US (2) | US10842853B2 (pt) |
EP (1) | EP3630974A1 (pt) |
JP (1) | JP6961171B2 (pt) |
KR (2) | KR102551733B1 (pt) |
CN (1) | CN110945127A (pt) |
AR (1) | AR112057A1 (pt) |
AU (1) | AU2018272831A1 (pt) |
BR (1) | BR112019024256A2 (pt) |
CA (1) | CA3064730A1 (pt) |
CL (2) | CL2019003395A1 (pt) |
CO (1) | CO2019012885A2 (pt) |
IL (2) | IL270567B (pt) |
MX (1) | MX2019013836A (pt) |
NZ (1) | NZ759034A (pt) |
TW (1) | TWI753168B (pt) |
WO (1) | WO2018217731A1 (pt) |
ZA (1) | ZA201907716B (pt) |
Families Citing this family (11)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
GB201420139D0 (en) | 2014-11-12 | 2014-12-24 | Ucl Business Plc | Factor IX gene therapy |
US10842885B2 (en) | 2018-08-20 | 2020-11-24 | Ucl Business Ltd | Factor IX encoding nucleotides |
US11596671B2 (en) | 2019-02-01 | 2023-03-07 | Takeda Pharmaceutical Company Limited | Gene therapy of hemophilia B using viral vectors encoding recombinant fix variants with increased expression |
WO2020187969A1 (en) * | 2019-03-19 | 2020-09-24 | CSL Behring Lengnau AG | Factor ix variants and uses thereof in therapy |
CN114375205A (zh) | 2019-06-20 | 2022-04-19 | 武田药品工业株式会社 | 用基于病毒的基因疗法进行治疗的方法 |
CN110684798A (zh) * | 2019-09-03 | 2020-01-14 | 深圳新诺微环生物科技有限公司 | 肌肉靶向的微环dna基因治疗 |
TW202246505A (zh) * | 2021-03-05 | 2022-12-01 | 俄羅斯聯邦商亞那拜恩有限公司 | 編碼凝血因子ix蛋白的密碼子優化的核酸及其用途 |
WO2022268110A1 (zh) * | 2021-06-23 | 2022-12-29 | 英斯培瑞有限公司 | 用于治疗b型血友病的组合物和方法 |
CN114277057B (zh) * | 2021-07-09 | 2023-10-13 | 上海天泽云泰生物医药有限公司 | 用于治疗或预防b型血友病的重组腺相关病毒载体和方法 |
CA3232470A1 (en) * | 2021-10-27 | 2023-05-04 | Leah SABIN | Compositions and methods for expressing factor ix for hemophilia b therapy |
WO2023202469A1 (zh) * | 2022-04-19 | 2023-10-26 | 康霖生物科技(杭州)有限公司 | 一种用于遗传性凝血因子缺乏病治疗的核酸构建体及其用途 |
Family Cites Families (32)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US4797368A (en) | 1985-03-15 | 1989-01-10 | The United States Of America As Represented By The Department Of Health And Human Services | Adeno-associated virus as eukaryotic expression vector |
US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
US6268213B1 (en) | 1992-06-03 | 2001-07-31 | Richard Jude Samulski | Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy |
JPH08511423A (ja) | 1993-06-10 | 1996-12-03 | ジェネティック セラピー,インコーポレイテッド | 血友病治療のためのアデノウイルスベクター |
US5786464C1 (en) | 1994-09-19 | 2012-04-24 | Gen Hospital Corp | Overexpression of mammalian and viral proteins |
CA2200835A1 (en) | 1994-09-23 | 1996-03-28 | Frederick M. Boyce | Use of a non-mammalian dna virus to express an exogenous gene in a mammalian cell |
US6013516A (en) | 1995-10-06 | 2000-01-11 | The Salk Institute For Biological Studies | Vector and method of use for nucleic acid delivery to non-dividing cells |
US5858351A (en) | 1996-01-18 | 1999-01-12 | Avigen, Inc. | Methods for delivering DNA to muscle cells using recombinant adeno-associated virus vectors |
US5866552A (en) | 1996-09-06 | 1999-02-02 | The Trustees Of The University Of Pennsylvania | Method for expressing a gene in the absence of an immune response |
US6114148C1 (en) | 1996-09-20 | 2012-05-01 | Gen Hospital Corp | High level expression of proteins |
WO1998041240A1 (en) | 1997-03-14 | 1998-09-24 | The Children's Hospital Of Philadelphia | Methods and compositions for use in gene therapy for treatment of hemophilia |
US6531298B2 (en) | 1997-07-21 | 2003-03-11 | The University Of North Carolina At Chapel Hill | Factor IX antihemophilic factor with increased clotting activity |
US5994136A (en) | 1997-12-12 | 1999-11-30 | Cell Genesys, Inc. | Method and means for producing high titer, safe, recombinant lentivirus vectors |
US6924365B1 (en) | 1998-09-29 | 2005-08-02 | Transkaryotic Therapies, Inc. | Optimized messenger RNA |
US8063022B1 (en) | 1999-06-08 | 2011-11-22 | The Children's Hospital Of Philadelphia | Methods for preventing formation of inhibitory antibodies in the setting of gene therapy |
CN1148228C (zh) | 2000-08-30 | 2004-05-05 | 夏家辉 | 治疗血友病b的基因药物及其制备方法 |
PT1453547T (pt) | 2001-12-17 | 2016-12-28 | Univ Pennsylvania | Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas |
WO2006036502A2 (en) | 2004-09-22 | 2006-04-06 | St. Jude Children's Research Hospital | Improved expression of factor ix in gene therapy vectors |
US7943374B2 (en) | 2005-08-21 | 2011-05-17 | Markus Hildinger | Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb |
WO2007149406A2 (en) * | 2006-06-19 | 2007-12-27 | Nautilus Technology Llc | Modified coagulation factor ix polypeptides and use thereof for treatment |
EP4154903A1 (en) | 2008-04-22 | 2023-03-29 | Vib Vzw | Liver-specific nucleic acid regulatory elements and methods and use thereof |
EP4219547A3 (en) | 2008-09-15 | 2023-10-18 | uniQure biopharma B.V. | Factor ix polypeptide mutant, its uses and a method for its production |
US9663778B2 (en) * | 2009-07-09 | 2017-05-30 | OPKO Biologies Ltd. | Long-acting coagulation factors and methods of producing same |
TWI557135B (zh) | 2010-11-03 | 2016-11-11 | 介控生化科技公司 | 經修飾之第九因子多胜肽及其用途 |
US10640785B2 (en) | 2011-11-22 | 2020-05-05 | The Children's Hospital Of Philadelphia | Virus vectors for highly efficient transgene delivery |
EP2791160B1 (en) * | 2011-12-16 | 2022-03-02 | ModernaTX, Inc. | Modified mrna compositions |
CA2864879C (en) | 2012-02-17 | 2021-07-20 | The Children's Hospital Of Philadelphia | Aav vector compositions and methods for gene transfer to cells, organs and tissues |
AU2013336601B2 (en) * | 2012-10-26 | 2018-01-25 | Vrije Universiteit Brussel | Vector for liver-directed gene therapy of hemophilia and methods and use thereof |
WO2014063753A1 (en) | 2012-10-26 | 2014-05-01 | Vrije Universiteit Brussel | Hyper-active factor ix vectors for liver-directed gene therapy of hemophilia 'b' and methods and use thereof |
EP3270944B1 (en) | 2015-03-17 | 2019-10-23 | Vrije Universiteit Brussel | Optimized liver-specific expression systems for fviii and fix |
KR102415896B1 (ko) | 2015-06-23 | 2022-06-30 | 더 칠드런스 호스피탈 오브 필라델피아 | 변형된 인자 ix, 및 세포, 기관 및 조직으로 유전자를 전달하기 위한 조성물, 방법 및 용도 |
CN116355883A (zh) * | 2016-10-14 | 2023-06-30 | 四川至善唯新生物科技有限公司 | 一种高活性凝血因子ix突变体、重组蛋白与融合蛋白的制备与应用 |
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2018
- 2018-05-22 EP EP18731636.9A patent/EP3630974A1/en active Pending
- 2018-05-22 AU AU2018272831A patent/AU2018272831A1/en active Pending
- 2018-05-22 TW TW107117388A patent/TWI753168B/zh active
- 2018-05-22 US US15/986,350 patent/US10842853B2/en active Active
- 2018-05-22 KR KR1020227012028A patent/KR102551733B1/ko active IP Right Grant
- 2018-05-22 MX MX2019013836A patent/MX2019013836A/es unknown
- 2018-05-22 BR BR112019024256A patent/BR112019024256A2/pt unknown
- 2018-05-22 AR ARP180101356A patent/AR112057A1/es unknown
- 2018-05-22 KR KR1020197037828A patent/KR102386890B1/ko active IP Right Grant
- 2018-05-22 CN CN201880042428.9A patent/CN110945127A/zh active Pending
- 2018-05-22 NZ NZ759034A patent/NZ759034A/en unknown
- 2018-05-22 WO PCT/US2018/033866 patent/WO2018217731A1/en unknown
- 2018-05-22 JP JP2019564430A patent/JP6961171B2/ja active Active
- 2018-05-22 CA CA3064730A patent/CA3064730A1/en active Pending
-
2019
- 2019-11-11 IL IL270567A patent/IL270567B/en active IP Right Grant
- 2019-11-18 CO CONC2019/0012885A patent/CO2019012885A2/es unknown
- 2019-11-21 CL CL2019003395A patent/CL2019003395A1/es unknown
- 2019-11-21 ZA ZA2019/07716A patent/ZA201907716B/en unknown
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2020
- 2020-09-23 CL CL2020002445A patent/CL2020002445A1/es unknown
- 2020-10-20 US US17/075,556 patent/US20210128700A1/en active Pending
-
2021
- 2021-02-04 IL IL280637A patent/IL280637A/en unknown
Also Published As
Publication number | Publication date |
---|---|
TWI753168B (zh) | 2022-01-21 |
KR102551733B1 (ko) | 2023-07-06 |
RU2019142702A3 (pt) | 2021-10-12 |
CN110945127A (zh) | 2020-03-31 |
KR20200010443A (ko) | 2020-01-30 |
NZ759034A (en) | 2023-07-28 |
TW201900184A (zh) | 2019-01-01 |
AR112057A1 (es) | 2019-09-18 |
CL2019003395A1 (es) | 2020-04-17 |
EP3630974A1 (en) | 2020-04-08 |
WO2018217731A1 (en) | 2018-11-29 |
IL280637A (en) | 2021-03-25 |
KR102386890B1 (ko) | 2022-04-15 |
JP2020520662A (ja) | 2020-07-16 |
US20180339026A1 (en) | 2018-11-29 |
JP6961171B2 (ja) | 2021-11-05 |
IL270567B (en) | 2021-03-25 |
KR20220050238A (ko) | 2022-04-22 |
ZA201907716B (en) | 2022-06-29 |
US20210128700A1 (en) | 2021-05-06 |
CO2019012885A2 (es) | 2020-01-17 |
CL2020002445A1 (es) | 2020-12-28 |
RU2019142702A (ru) | 2021-06-24 |
MX2019013836A (es) | 2020-01-30 |
AU2018272831A1 (en) | 2019-11-28 |
CA3064730A1 (en) | 2018-11-29 |
US10842853B2 (en) | 2020-11-24 |
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